Gene therapy aims to halt blindness from rare eye disease
NCT ID NCT03293524
First seen Apr 30, 2026 · Last updated May 16, 2026 · Updated 4 times
Summary
This study tested a gene therapy called GS010 in 98 people with Leber hereditary optic neuropathy (LHON), a genetic condition that causes sudden vision loss. Participants received injections into both eyes and were followed for up to 5 years. The goal was to see if the treatment could improve or stabilize eyesight compared to a placebo.
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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CHNO Les Quinze Vingts
Paris, Paris, 75012, France
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Department of Ophthalmology, Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
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Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University
Philadelphia, Pennsylvania, 19107, United States
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Doheny Eye Center UCLA Pasadena
Pasadena, California, 91105, United States
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Emory Healthcare - The Emory Clinic
Atlanta, Georgia, 30322, United States
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Hospital Universitario Ramon y Cajal
Madrid, 28034, Spain
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IRCCS Istituto delle Scienze Neurologiche di Bologna (ISNB) Clinica Neurologica
Bologna, 40139, Italy
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Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114, United States
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Moorfields Eye Hospital
London, Greater London, EC1V 2PD, United Kingdom
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Taipei Veterans General Hospital
Taipei, 11217, Taiwan
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Universitair Ziekenhuis Gent
Ghent, 9000, Belgium
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University of Colorado Health Eye Center
Aurora, Colorado, 80045, United States
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Vanderbilt Eye Institute
Nashville, Tennessee, 37232, United States
Conditions
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